Study to Assess the Efficacy and Safety of Omalizumab Treatment on ICS Reduction for Severe IgE-mediated Asthma

Phase 4

Terminated

Conditions: Severe IgE-mediated Asthma

Interventions: Drug: Omalizumab
Drug: Budesonide
Drug: Formoterol

Registration Number: NCT01912872

Lead Sponsor: Novartis Pharmaceuticals

Brief Summary: Assess efficacy and safety of omalizumab treatment during 12 months in order to reduce the use of inhaled corticosteroid (ICS) in pediatric and adult participants with severe Immunoglobulin E (IgE)-mediated asthma inadequately controlled with high doses of corticosteroids.

Detailed Description: This was a multicentric, open label, randomized, parallel-group study with a 12-month treatment period. Participants were assigned to one of the 2 treatment groups, omalizumab plus budesonide/formoterol or budesonide/formoterol alone.

The study comprised 4 phases:

During the 4-week run-in phase adult participants received budesonide 800 mg and formoterol 24 mg. If a participant complied with all inclusion and exclusion criteria and had received the according-to-age run-in proposed doses during the last month, the participant continued to the stable-steroid phase.

During the 16-week stable-steroid phase, adult and pediatric eligible participants were randomized to one of the two treatment groups.

During the 8-week steroid-reduction phase, adult and pediatric participants reduced 25% of the budesonide baseline dose every 2 weeks, depending of the asthma control, until they reached a 100% reduction of the baseline dose. The clinical control of asthma was defined according to criteria (GINA 2012).

Recruitment & Eligibility

Status: TERMINATED

Sex: All

Target Recruitment: 112

Inclusion Criteria

Not provided

Exclusion Criteria

Not provided

Study & Design

Study Type: INTERVENTIONAL

Study Design: PARALLEL

Arm && Interventions

Group	Intervention	Description
Omalizumab + budesonide and formoterol	Omalizumab	Participants will receive Omalizumab every 2 or 4 weeks depending on IgE level and body weight and will also receive budesonide and formoterol according to maximum daily dose.
Omalizumab + budesonide and formoterol	Budesonide	Participants will receive Omalizumab every 2 or 4 weeks depending on IgE level and body weight and will also receive budesonide and formoterol according to maximum daily dose.
Budesonide and formoterol	Budesonide	Participants will receive budesonide and formoterol according to maximum daily dose.
Omalizumab + budesonide and formoterol	Formoterol	Participants will receive Omalizumab every 2 or 4 weeks depending on IgE level and body weight and will also receive budesonide and formoterol according to maximum daily dose.
Budesonide and formoterol	Formoterol	Participants will receive budesonide and formoterol according to maximum daily dose.

Primary Outcome Measures

Name	Time	Method
The Mean Prescribed Budesonide Dose (μg) at Baseline	Baseline	prescribed budesonide dose (in μg) at Baseline in intention to treat population and in intention to treat population

Secondary Outcome Measures

Name	Time	Method
Number of Hospital Admissions Due to Asthma Exacerbation	12 month treatment duration	A hospital admission is defined as admissions to hospital involving a stay of at least 24 hours.
Participants Requiring Oral Systemic Corticosteroids During the 12 Month Study Duration	12 month treatment duration	Number of days of concomitant medications use reported by participants at all visits via diaries.
Control of Asthma Symptoms- Daytime Symptoms	12 month treatment duration	The clinical control of asthma was defined according to the following criteria (GINA 2012): 1-Daytime symptoms: none or less than twice a week 2-Limitations of daily activities: none 3-Nocturnal symptoms or awakening because of asthma: none 4-Need of relief or rescue medication: none or less than twice a week 5-Lung function (PEF or FEV1) without administration of bronchodilator: normal
Days Missed in School/Work Due to Asthma Exacerbation Episodes	12 month treatment duration	Participants /parent/legal guarding reported number of missed days of school or work at each study visit via diaries.
Control of Asthma Symptoms	12 month treatment duration	The clinical control of asthma was defined according to the following criteria (GINA 2012): 1-Daytime symptoms: none or less than twice a week 2-Limitations of daily activities: none 3-Nocturnal symptoms or awakening because of asthma: none 4-Need of relief or rescue medication: none or less than twice a week 5-Lung function (PEF or FEV1) without administration of bronchodilator: normal
Control of Asthma Symptoms- Rescue Medication Use	12 month treatment duration	The clinical control of asthma was defined according to the following criteria (GINA 2012): 1-Daytime symptoms: none or less than twice a week 2-Limitations of daily activities: none 3-Nocturnal symptoms or awakening because of asthma: none 4-Need of relief or rescue medication: none or less than twice a week 5-Lung function (PEF or FEV1) without administration of bronchodilator: normal
Asthma Control Questionnaire (ACQ) at Baseline	Baseline	The Asthma Control Questionnaire (ACQ) has six questions to be answered by the participants, each with a 7 point scale (0-good control, 6-poor control), and one question where the actual pre-bronchodilator Forced expiratory volume in 1 second (FEV1) value expressed in % of predicted FEV1 was classified to scores from 0 (\> 95% of predicted) to 6 (\< 50% of predicted). The overall score is the average of the 7 questions; a minimum overall score of 0 = good control of asthma whereas a maximum overall score of 6 = poor control of asthma.
Asthma Quality of Life Questionnaire (AQLQ) at Baseline	Baseline	The quality of life will be measured by the standardized version of the Asthma Quality of Life Questionnaire (AQLQ\[S\]) score for adults and the pediatric version of the AQLQ(S) for pediatric participants (PAQLQ\[S\]) . The AQLQ(S) and PAQLQ(S0 contain 4 domains (activity limitations, symptoms, emotional function, and environmental stimuli), with a total of 32 items; each item is measured in a 7-point Likert scale of 1 to 7 (1 = severe impairment, 7 = no impairment). All items are weighted equally. Mean score is calculated across all items within each domain and the overall score is the mean score of the 32 items.